Efficacy, safety, and regulatory approval of FDA–designated breakthrough and non-breakthrough cancer medicines

J Clin Oncol

24 April 2018 - The breakthrough therapy program was established in 2012 to expedite the development and review of new medicines. Hwang et al. evaluated the times to approval, efficacy, and safety of breakthrough-designated versus non–breakthrough-designated cancer drugs approved by the US FDA.

They studied all new cancer drugs approved by the FDA between January 2012 and December 2017. Regulatory and therapeutic characteristics (time to FDA approval, pivotal trial efficacy end point, novelty of mechanism of action) were compared between breakthrough-designated and non–breakthrough-designated cancer drugs. Random-effects meta-regression was used to assess the association between breakthrough therapy designation and hazard ratios for progression-free survival, response rates  for solid tumours, serious adverse events, and deaths not attributed to disease progression.

Between 2012 and 2017, the FDA approved 58 new cancer drugs, 25 (43%) of which received breakthrough therapy designation. The median time to first FDA approval was 5.2 years for breakthrough-designated drugs versus 7.1 years for non–breakthrough-designated drugs (difference, 1.9 years; P = .01).

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Michael Wonder

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Michael Wonder